FDA Issues Draft Guidance for ALS Clinical Trials

This release is part of a larger effort to update the FDA's new drug review programs

Published

Friday, February 16, 2018

Author

Robert Goldstein

This week, the Food and Drug Administration (FDA) issued a draft guidance for those developing potential treatments for ALS. This document, which can be read online here, is part of an effort by the FDA to make drug developers and others aware of how they think clinical trials in ALS should be run. This is the first time the FDA has issued a guidance for industry on ALS clinical trials.

“We hope that providing up-to-date, clear information about our scientific expectations, such as clinical trial design and ways to measure effectiveness, will save companies time and resources and ultimately, bring effective new medicines to patients more efficiently,” said FDA Commissioner Scott Gottlieb, M.D.

Right now, big pharma and small biotech companies, as well as academic investigators alike, are testing two dozen different proposed treatments in people with ALS. Well over 2,000 people living with ALS have or will enroll in one of those trials in the USA alone. While the draft guidance is unlikely to impact the design of trials currently underway, it will certainly impact those put before the FDA for approval in the future. That document can be read and commented upon here.

The ALS Therapy Development Institute (ALS TDI) will hold a public webinar on the topic of how drugs are approved by the FDA on March 8, 2018. This guidance will be reviewed in that webinar, among other topics. Registration is required in advance here.

Robert Goldstein

I have been working at ALS TDI since 2004. I write about the latest science in ALS and aim to provide a layman's overview of topics important to patients, caregivers, clinicians and researchers alike. I am a Staff Writer and Editor for our Research News and Commentary sections. Email me: rgoldstein@als.net.